Leslie B Gordon
Affiliation: Brown University
- Inhibiting farnesylation of progerin prevents the characteristic nuclear blebbing of Hutchinson-Gilford progeria syndromeBrian C Capell
Genome Technology Branch, National Human Genome Research Institute, National Institutes of Health, 50 South Drive, MSC 8004, Bethesda, MD 20892 8004, USA
Proc Natl Acad Sci U S A 102:12879-84. 2005..Last, treatment of both early- and late-passage human HGPS fibroblasts with FTIs resulted in significant reductions in nuclear blebbing. Our results suggest that treatment with FTIs represents a potential therapy for patients with HGPS...
- Hutchinson-Gilford progeria mutant lamin A primarily targets human vascular cells as detected by an anti-Lamin A G608G antibodyDayle McClintock
Department of Dermatology, College of Physicians and Surgeons, Columbia University, New York, NY 10032, USA
Proc Natl Acad Sci U S A 103:2154-9. 2006..This finding suggests that accumulation of progerin is directly involved in vascular disease in progeria...
- Age-dependent loss of MMP-3 in Hutchinson-Gilford progeria syndromeIngrid A Harten
Hope Heart Matrix Biology Program, Benaroya Research Institute at Virginia Mason, Seattle, Washington, USA
J Gerontol A Biol Sci Med Sci 66:1201-7. 2011..This metalloproteinase has the potential to serve as a biomarker of therapeutic efficacy when assessing treatments for HGPS...
- Clinical Trial of the Protein Farnesylation Inhibitors Lonafarnib, Pravastatin, and Zoledronic Acid in Children With Hutchinson-Gilford Progeria SyndromeLeslie B Gordon
From Departments of Anesthesia L B G, M E K, A A D, K L, M M G, Cardiology L B S, Radiology R H C, V M S, Orthopedics B D S, Neurology N J U, Dermatology M G L, Genetics and Genomics D T M, Gastroenterology and Nutrition S Y H, and Hematology Oncology M W K, and Clinical Translational Study Unit N Q, Boston Children s Hospital and Harvard Medical School, MA Department of Pediatrics, Hasbro Children s Hospital and Warren Alpert Medical School of Brown University, Providence, RI L B G Department of Biostatistics, Boston University School of Public Health and Harvard Clinical Research Institute, MA J M, R B D, H S Division of Cardiology, Brigham and Women s Hospital and Harvard Medical School, Boston, MA M G H Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati Children s Hospital Medical Center, OH C M G Center for Advanced Orthopaedic Studies, Department of Orthopedic Surgery, Beth Israel Deaconess Medical Center and Harvard Medical School, Boston, MA A N and Division of Pediatric Oncology, Geneva
Circulation 134:114-25. 2016..This present trial sought to further improve disease by additionally inhibiting progerin prenylation...
- Impact of farnesylation inhibitors on survival in Hutchinson-Gilford progeria syndromeLeslie B Gordon
From the Department of Pediatrics, Hasbro Children s Hospital and Warren Alpert Medical School of Brown University, Providence, RI L B G Department of Anesthesia, Division of Critical Care Medicine, Boston Children s Hospital and Harvard Medical School, Boston, MA L B G, M E K Department of Mathematics and Statistics, Boston University, Harvard Clinical Research Institute, Boston, MA J M, R B D Center for Gerontology and Health Care Research, Brown University, Providence, RI S E C, J B Department of Genetics, New York State Institute for Basic Research, Staten Island, NY W T B Hematology Oncology, Boston Children s Hospital, Division of Pediatric Oncology, Dana Farber Cancer Institute and Harvard Medical School, Boston, MA M W K
Circulation 130:27-34. 2014..The key elements necessary for this analysis are a robust natural history of survival and comparison with a sufficiently large patient population that has been treated for a sufficient time period with disease-targeting medications...
- Progeria: a paradigm for translational medicineLeslie B Gordon
Department of Anesthesia, Boston Children s Hospital and Harvard Medical School, Boston, MA 02115, USA Department of Pediatrics, Hasbro Children s Hospital and Warren Alpert Medical School of Brown University, Providence, RI 02912, USA Electronic address
Cell 156:400-7. 2014..The progress made on the premature aging disorder Progeria is a shining example of the impact that studies of rare diseases can have. ..
- Clinical trial of a farnesyltransferase inhibitor in children with Hutchinson-Gilford progeria syndromeLeslie B Gordon
Department of Anesthesia, Boston Children s Hospital and Harvard Medical School, Boston, MA 02115, USA
Proc Natl Acad Sci U S A 109:16666-71. 2012..Results from this clinical treatment trial for children with HGPS provide preliminary evidence that lonafarnib may improve vascular stiffness, bone structure, and audiological status...
- Highlights of the 2007 Progeria Research Foundation scientific workshop: progress in translational scienceLeslie B Gordon
Warren Alpert Medical School of Brown University, Providence, RI 02912, USA
J Gerontol A Biol Sci Med Sci 63:777-87. 2008
- Disease progression in Hutchinson-Gilford progeria syndrome: impact on growth and developmentLeslie B Gordon
Department of Pediatrics, Rhode Island Hospital, Providence, Rhode Island, USA
Pediatrics 120:824-33. 2007..We sought to more clearly define the bone and weight abnormalities in patients with progeria as potential outcome parameters for prospective clinical trials...
- A prospective study of radiographic manifestations in Hutchinson-Gilford progeria syndromeRobert H Cleveland
Pediatric Radiology, Children s Hospital Boston, Harvard Medical School, Boston, MA 02115, USA
Pediatr Radiol 42:1089-98. 2012..There has been no comprehensive prospective study describing the skeletal abnormalities associated with progeria...
- Mechanisms of premature vascular aging in children with Hutchinson-Gilford progeria syndromeMarie Gerhard-Herman
Division of Cardiology, Brigham and Women s Hospital and Harvard Medical School, Boston, MA, USA
Hypertension 59:92-7. 2012....
- Neurologic features of Hutchinson-Gilford progeria syndrome after lonafarnib treatmentNicole J Ullrich
Department of Neurology, Boston Children s Hospital and Harvard Medical School, Boston, MA, USA
Neurology 81:427-30. 2013....
- Hutchinson-Gilford progeria is a skeletal dysplasiaCatherine M Gordon
Division of Adolescent Medicine and Endocrinology, Children s Hospital Boston, Harvard Medical School, Boston, MA, USA
J Bone Miner Res 26:1670-9. 2011..Dietary intake was adequate, confirming that HGPS does not represent a model of malnutrition-induced bone loss. Taken together, these findings suggest that the phenotype of HGPS represents a unique skeletal dysplasia...
- Recurrent de novo point mutations in lamin A cause Hutchinson-Gilford progeria syndromeMaria Eriksson
Department of Human Genetics, New York State Institute for Basic Research in Developmental Disabilities, Staten Island, New York 10314, USA
Nature 423:293-8. 2003..The discovery of the molecular basis of this disease may shed light on the general phenomenon of human ageing...
- Aggrecan expression is substantially and abnormally upregulated in Hutchinson-Gilford Progeria Syndrome dermal fibroblastsJoan M Lemire
Department of Anatomy and Cellular Biology, Tufts University School of Medicine, Boston, MA, USA
Mech Ageing Dev 127:660-9. 2006..This demonstrates that elevated aggrecan expression and its secretion are aberrant features of HGPS. We conclude that HGPS cells can display massively altered transcript levels leading to the secretion of inappropriate protein species...
- Initial cutaneous manifestations of Hutchinson-Gilford progeria syndromeJillian F Rork
Department of Dermatology, Boston Children s Hospital, Harvard Medical School, Boston, Massachusetts
Pediatr Dermatol 31:196-202. 2014..HGPS has distinct cutaneous manifestations during the first 2 years of life that may be the first signs of disease. Awareness of these findings could expedite diagnosis...
- Reduced adiponectin and HDL cholesterol without elevated C-reactive protein: clues to the biology of premature atherosclerosis in Hutchinson-Gilford Progeria SyndromeLeslie B Gordon
Department of Anatomy and Cellular Biology, Tufts University School of Medicine, Boston, MA 02111, USA
J Pediatr 146:336-41. 2005..We determined whether children with HGPS demonstrate abnormalities in known biomarkers for cardiovascular disease (CVD) risk...
- Hutchinson-Gilford progeria syndromeNicole J Ullrich
Department of Neurology, Boston Children s Hospital and Harvard Medical School, Boston, MA, USA
Handb Clin Neurol 132:249-64. 2015..This chapter will discuss both preclinical and clinical aspects of the genetics, pathobiology, clinical phenotype, clinical care, and treatment of HGPS, with special attention toward neurologic and cutaneous findings. ..
- Hyaluronan is not elevated in urine or serum in Hutchinson-Gilford Progeria SyndromeLeslie B Gordon
Department of Anatomy and Cellular Biology, Tufts University School of Medicine, 136 Harrison Avenue, Boston, MA 02111, USA
Hum Genet 113:178-87. 2003..These studies indicate that neither serum nor urinary hyaluronan concentration is a reliable diagnostic or prognostic marker for HGPS and underscore a difference between adult and childhood progerias...
- Accumulation of mutant lamin A causes progressive changes in nuclear architecture in Hutchinson-Gilford progeria syndromeRobert D Goldman
Department of Cell and Molecular Biology, Northwestern University Feinberg School of Medicine, 303 East Chicago Avenue, Chicago, IL 60611, USA
Proc Natl Acad Sci U S A 101:8963-8. 2004....
- Progressive vascular smooth muscle cell defects in a mouse model of Hutchinson-Gilford progeria syndromeRenee Varga
Genome Technology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA
Proc Natl Acad Sci U S A 103:3250-5. 2006..This mouse model should prove valuable for testing experimental therapies for this devastating disorder and for exploring cardiovascular disease in general...
- The mutant form of lamin A that causes Hutchinson-Gilford progeria is a biomarker of cellular aging in human skinDayle McClintock
Department of Dermatology, College of Physicians and Surgeons, Columbia University, New York, New York, United States of America
PLoS ONE 2:e1269. 2007..Our findings demonstrate that progerin expression is a biomarker of normal cellular aging and may potentially be linked to terminal differentiation and senescence in elderly individuals...
- Phenotype and course of Hutchinson-Gilford progeria syndromeMelissa A Merideth
National Human Genome Research Institute, Intramural Office of Rare Disease, National Institutes of Health, Bethesda, MD 20892 1851, USA
N Engl J Med 358:592-604. 2008....
- Predictors of acquired lipodystrophy in juvenile-onset dermatomyositis and a gradient of severityApril Bingham
From Office of Clinical Research, National Institute of Environmental Health Sciences, National Institutes of Health, Bethesda, Maryland 20892 1301, USA
Medicine (Baltimore) 87:70-86. 2008..Further study is warranted to investigate the pathogenesis of acquired LD in patients with DM...